Latest News: Research
18 October 2017 / Posted in: Research
We share the frustrations of the SMA Community that there has still been no announcement from NICE about whether it will assess the treatment for continued and wider provision of nusinersen by the NHS. Here is an update of what SMA Support UK is doing.
12 October 2017 / Posted in: Research
The Food and Drug Administration (FDA) has granted permission to AveXis to begin their planned US pivotal trial of their gene therapy drug for SMA, AVXS-101. Avexis say that they are to immediately begin the trial.
11 October 2017 / Posted in: Research
Muscular Dystrophy UK are campaigning for faster access to specialist treatments, including Spinraza for SMA. You can find out more and support the 'FastTrack' Campaign here.
29 September 2017 / Posted in: Research
The request to include those with more than two SMN2 copies and over 7 months of age in the SMA Type 1 nusinersen EAP is not yet successful. We are still waiting to hear from NICE about next steps.
27 September 2017 / Posted in: Research
Novartis has released the following community update for the LMI070 (branaplam) clinical trial.
19 September 2017 / Posted in: Research
Roche have provided an update on clinical trials of their SMN2 splice-modifying drug RG7916
30 August 2017 / Posted in: Research
You can now read the full workshop report written by the clinical researchers and patient representatives from Europe and the US who met last year.
24 August 2017 / Posted in: Research
Sent on 23rd August from clinicians and advocacy groups, this asks for the age limit and SMN2 copy number restrictions on potential access to be removed.
16 August 2017 / Posted in: Research
Roche has begun to enrol SMA patients in the FIREFISH Phase II trial of their splice-modifying drug, RG7916 (also known as RO7034067).
07 August 2017 / Posted in: Research
We have clarified that, from now on going forwards (until we hear from NICE), children will have to be diagnosed by 7 months of age to be eligible. If you have a child who has already been diagnosed with SMA Type 1 who is older than this and not already on the EAP, please read more.